Cappelli Francesco, Ponti Lucia, Hsu Kristen, Damy Thibaud, Villacorta Eduardo, Verheyen Nicolas, Keohane Denis, Wang Ronnie, Ines Monica, Kumar Nisith, Munteanu Carmen
Tuscan Regional Amyloidosis Referral Centre, Careggi University Hospital, Florence, Italy.
University of Urbino, Urbino, Italy.
Front Cardiovasc Med. 2025 Jun 9;12:1595797. doi: 10.3389/fcvm.2025.1595797. eCollection 2025.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive condition with debilitating symptoms. The self-reported burden of ATTR-CM on patients and their caregivers was previously evaluated in an international, multicenter, real-world survey study.
This analysis used univariate and multivariate models to evaluate survey items as predictors of ATTR-CM burden. The final multivariate models were optimized using forward selection and CV Press criteria with 8-fold cross-validation to include only the best predictors. Hierarchical linear regression analyses were used to explore potential moderators of the relationship between patient health status and caregiver burden.
The original survey included 208 patients with ATTR-CM, naïve to disease-modifying treatment, and their unpaid primary caregivers from international amyloidosis centers of excellence in 7 countries between July 2021 and August 2022. Most patients were male (86%), elderly (median age, 81 years), and had untreated wild-type ATTR-CM (91% of 155 with genetic testing). Patients reported fair to good health status overall [Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) median score, 68]. Most (60%) of the 199 patients with a New York Heart Association (NYHA) classification were class II (18% class I; 22% class III). Optimized multivariate models for several measures found symptomatic heart failure (NYHA class II or III), having "weakness, especially in the legs," older age, and female sex, were independent predictors of higher patient-reported burden. The majority of caregivers were female (85%) and the spouse (59%) or adult child (37%) of the patient. The median duration of caregiving was 1.5 years. In the final optimized multivariate model, only the patient's KCCQ-OS score was a significant predictor of caregiver burden. This relationship was not clinically moderated by other patient or caregiver variables.
Our analysis showed that heart failure symptoms, weakness, especially in the legs, older age, and female sex, are independent predictors of higher disease burden in patients with ATTR-CM. A higher caregiver burden was best predicted by poorer health status in the patient, even in the presence of potential moderators. Implementing strategies to reduce the physical symptoms experienced by patients with ATTR-CM may help to reduce their burden, and that experienced by caregivers.
转甲状腺素蛋白淀粉样变心肌病（ATTR-CM）是一种症状逐渐加重的疾病。此前，在一项国际多中心真实世界调查研究中评估了ATTR-CM对患者及其照护者的自我报告负担。
本分析使用单变量和多变量模型来评估调查项目，作为ATTR-CM负担的预测因素。最终的多变量模型使用向前选择和CV Press标准以及8折交叉验证进行优化，以仅纳入最佳预测因素。使用分层线性回归分析来探索患者健康状况与照护者负担之间关系的潜在调节因素。
原始调查纳入了208例未接受疾病修饰治疗的ATTR-CM患者及其 unpaid 主要照护者，这些患者来自2021年7月至2022年8月期间7个国家的国际淀粉样变性卓越中心。大多数患者为男性（86%），年龄较大（中位年龄81岁），且患有未经治疗的野生型ATTR-CM（155例接受基因检测的患者中91%）。患者总体报告健康状况为中等至良好[堪萨斯城心肌病问卷总体总结（KCCQ-OS）中位得分68]。199例纽约心脏协会（NYHA）分级患者中，大多数（60%）为II级（I级18%；III级22%）。针对多项指标的优化多变量模型发现，有症状的心力衰竭（NYHA II级或III级）、“虚弱，尤其是腿部”、年龄较大和女性是患者报告的较高负担的独立预测因素。大多数照护者为女性（85%），是患者的配偶（59%）或成年子女（37%）。中位照护时长为1.5年。在最终优化的多变量模型中，只有患者的KCCQ-OS得分是照护者负担的显著预测因素。这种关系未受到其他患者或照护者变量的临床调节。
我们的分析表明，心力衰竭症状、虚弱，尤其是腿部、年龄较大和女性是ATTR-CM患者疾病负担较高的独立预测因素。即使存在潜在调节因素，患者健康状况较差也最能预测照护者负担较高。实施策略减轻ATTR-CM患者经历的身体症状可能有助于减轻他们及其照护者的负担。
